ALPMY - FDA lifts clinical hold on Astellas' gene replacement therapy trial

Astellas Pharma ( OTCPK:ALPMY ) ( OTCPK:ALPMF ) said the U.S. Food and Drug Administration (FDA) lifted a clinical hold on a trial of its gene replacement therapy AT845 to treat adults with late-onset Pompe disease (LOPD).

The FDA had placed a hold on the phase 1/2 trial, dubbed FORTIS, in June 2022 after a serious adverse event (SAE) of damage to peripheral nerves in one patient was reported.

The Japanese company said on Friday that following the lifting of this clinical hold, it is working on completing the clinical and regulatory activities required to resume dosing in the study.

Astellas noted that the clinical hold lift will have no impact on its financial forecasts of the current fiscal year ending March 31.

The study is evaluating AT845, an investigational adeno-associated virus (AAV) gene replacement therapy in adults with LOPD — a rare genetic disorder is characterized by skeletal muscle weakness affecting mobility and the respiratory system.

The disease is caused by mutations in the acid alpha-glucosidase (GAA) gene which prevent the production and function of a protein called GAA. AT845 is being investigated to determine if it can deliver a functional GAA gene which is efficiently transduced to express GAA directly in tissues, according to the company.

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